Acromegaly Update

A thin week: no top-tier-journal acromegaly papers appeared, so Part A draws on the next tier (a radiomics model to predict somatostatin-analog response, a familial Carney-complex case, and bone proteomics), while the rare disease industry's big story is uniQure's four-year Huntington's gene therapy data, which showed a smaller, non-significant benefit and sent the stock down about 40%.

A quiet week for top-tier acromegaly literature (four papers, mostly real-world/consensus rather than trial data) paired with a livelier rare disease industry week — a first-ever positive pivotal trial in X-linked retinitis pigmentosa and a new FOP approval that goes head-to-head with Regeneron — while Marea's own MAR002 acromegaly program remains the clearest bridge between the two segments following its merger with Lisata.

This week's acromegaly literature centers on long-term disease burden — cardiovascular morbidity, musculoskeletal disease, and precision-medicine treatment selection — while the rare disease industry notched a landmark ultra-rare gene therapy approval, a $1.65B radiopharma merger, and a $123M bet on an oral thyroid eye disease pill.

It's a quiet week for acromegaly-specific research — just one review paper worth flagging — but rare disease industry news was busy, headlined by Scholar Rock's first-ever muscle-targeted SMA approval and a rough week for two RNA-based rare disease programs at Novartis.

It's a thin week for acromegaly-specific research — no top-tier-journal hits, so we're covering the best next-tier evidence on GH-axis physiology, octreotide drug delivery, and pituitary imaging AI — while rare disease industry news delivers a sharp one-two: Ionis just won the first-ever approval for Alexander disease, days after Ultragenyx's marquee Angelman syndrome drug flopped in Phase 3.